Pipeline – Overview of the target diseases

Our company carefully evaluates and selects target diseases for exosome (EV) formulations.

Disease Overview[1]	ARDS is a severe respiratory failure caused by acute lung injury. Symptoms include sudden breathlessness, difficulty breathing, and severe shortness of breath. Mortality rate is 35-46%.[1] Treatment involves respiratory management, such as oxygen therapy and mechanical ventilation, and drug therapy. However, current pharmacological interventions, including steroids, have limited efficacy and lack strong evidence for improving survival rates.[3] Therefore, there is a pressing need for the development of new, effective drugs that can significantly reduce mortality and improve patient outcomes in ARDS.
Market Size[2]	The global market for ARDS therapeutics is projected to reach approximately $3.7 billion in 2024 and $4.9 billion in 2029, with an average annual growth rate of 5.7%.[2]
Reasons for Targeting	ARDS has a high mortality rate, but no drugs significantly improve survival. [3] Mesenchymal stem cells (MSCs) are known to have anti-inflammatory effects and have shown potential in improving ARDS survival rates in some clinical trials. Our MSC-derived EXP01 demonstrated higher anti-inflammatory effects in disease model mice compared to using MSCs as intact cells. Given the lack of effective treatments and the promising results of MSC-based clinical trials, targeting ARDS with EXP01 presents a significant opportunity to win the market.

Disease Overview[4]	IPF is a progressive disease causing lung fibrosis and difficulty breathing. Median survival after diagnosis is 3-5 years. Approved anti-fibrotic drugs have significant side effects.[1,4]
Market Size[2]	The global market for IPF therapeutics is expected to grow from $5.2 billion in 2024 to $7.3 billion by 2029, with an average annual growth rate of 7.0%.[6]
Reasons for Targeting	Current IPF treatments, such as pirfenidone and nintedanib, have limited efficacy and significant side effects, leading to poor patient outcomes. MSCs have demonstrated potential in reducing inflammation and fibrosis, showing promise in clinical trials. Our candidate EXP01, derived from MSCs using our proprietary technology, has shown superior anti-fibrotic effects in preclinical studies. By targeting IPF with EXP01, we aim to provide a more effective and safer therapeutic option to address the unmet needs of IPF patients.

Background and Solutions	Globally, infertility affects approximately 1 in 6 couples.Causes include age-related decline in egg quality/quantity. We are developing EXP02, derived from MSCs, which promotes germ cell development specifically for treating premature ovarian failure (POF).
Market Size[6]	The global market for infertility drugs is projected to reach a cumulative $4.1 billion by 2022 and expand to $7.2 billion by 2032, with an average annual growth rate of 5.9%.[6]
POF Overview[7] and Targeting	POF causes premature menopause and infertility, affecting 1% under 40.[7] Currently, no curative treatments exist. EXP02 has shown potential in preclinical studies by improving ovarian function in mice, demonstrating its promise as a therapeutic option for POF.

[1] The Japanese Respiratory Society, Japan Intractable Diseases Information Center, Crit Care. 2023 27:31
[2] Mordor intelligence 2023
[3] ARDS Clinical Practice Guidelines 2021
[4] The Japanese Respiratory Society, Japan Intractable Diseases Information Center
[5] Boston Consulting Group estimate (Ministry of Economy, Trade and Industry FY2023 Healthcare Industry Infrastructure Enhancement Promotion Project)
[6] Global Market Insights 2023
[7] The Japan Endocrine Society